Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia

NCT05773729 | Recruiting

• 1 other identifier: BD-TDT-211003
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.

Conditions

β-thalassemia

Outcome Measures

Primary Outcomes (1)

• Percentage of treated participants with Transfusion-Dependent β-Thalassemia (TDT) who achieved transfusion independence (TI) for at least 6 months

  TI defined as peripheral blood weighted average hemoglobin (Hb) \> or = 9 g/dL without packed red blood cell (pRBC) transfusion for 60 days after BD211 treatment, and transfusion is continuously halted for 12 months.

  24 months

Secondary Outcomes (14)

• Hb (g/dL) level between 12 and 24 months after BD211 treatment compared with baseline Hb level

  24 months

• Parameters of efficacy related to TI achievement after BD211 treatment

  24 months

• Parameters of efficacy related to reduced blood transfusion after BD211 treatment

  24 months

• Parameters of iron overload after BD211 treatment

  24 months

• Parameters of growth and development after BD211 treatment

  24 months

Study Arms (1)

BD211 Single-Dose group

EXPERIMENTAL

Route of Administrate: infusion intravenously. Dosage form: injection solution. Dose: 5×10\^6 cells /kg \~ 10×10\^6 cells /kg. Frequency of administration: One dosing intravenously. Intervention: Single dose of BD211

Genetic: BD211

Interventions

BD211 GENETIC

Genetically modified CD34+ autologous stem cells were transfused intravenously with single dosing.

BD211 Single-Dose group

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Ages 3 to 18 years old, including:
• The parents or legal guardians must be able to understand and provide ICFs. If available, it is strongly recommended that children aged ≥8 years in treatment decisions and obtain written ICFs and be clearly documented; Diagnosed as Transfusion Dependent β-thalassemia with any genotype (β0, β+, βE/β0, βS/S, βS/β0, βS/β+), confirmed the Hb analysis. No alfa chain genetic abnormalities. Subjects must stabilize and maintain an appropriate iron chelation regimen. Transfusion-dependent types are defined as requiring at least 100 mL/kg/ year of red blood cells (pRBCs).
• No eligiblity for allogeneic hematopoietic stem cell transplantation.
• The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.
• The subjects' parents/legal guardians must be willing and able to follow the study procedures in the study protocol.
• Good organs' functions.
• Having complete medical records including a history of blood transfusions testified subject received treatment and followed up for at least two years prior to screening .

You may not qualify if:

• HIV-1 and HIV-2 were positive, and / or HTLV-1, HTLV-2 and VSV-G antibodies were positive.
• An active bacterial, viral, fungal or parasitic infection.
• Contraindicated for the extraction of bone marrow under anesthesia.
• Any malignancy, myeloproliferative, or immunodeficient disease and relevant medical history.
• Peripheral blood white blood cell (WBC) count \< 3×10\^9/L or platelet count \< 120×10\^9/L.
• A history of allo-transplantation.
• Erythropoietin was used within 3 months prior to HSC cell collection.
• Immediate family members with known or suspected familial cancer syndromes (including but not limited to breast, colorectal, ovarian, prostate, and pancreatic cancers).
• Subjects with a diagnosis of major mental illness may had a serious disability to participate in the study.
• Active recurrent malaria.
• Had autoimmune diseases that may make blood transfusions difficult.
• History of major organ injury including:
• There are bleeding diseases that have not been cured.
• The subject involved with another clinical study in a 30-day screening period.
• Allergic to the research drug and its excipients.

Sponsors & Collaborators

• Shanghai BDgene Co., Ltd.: lead
• Shanghai Children's Medical Center: collaborator

Study Sites (1)

Shanghai Children's Medical Centre

Shanghai, 200127, China

RECRUITING

Study Officials

• Chen Jing, M.D.

  Shanghai Children's Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chen Jing, M.D.

CONTACT

+86-18930830632; chenjing@scmc.com.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 3, 2023
• First Posted: March 17, 2023
• Study Start: September 15, 2023
• Primary Completion: March 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: June 14, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)